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Currently, the indications used for MA (Marketing Authorization) Cinacalcet in France are hyperparathyroidism (hyperPTH) in adults, whether primary (for patients in whom parathyroidectomy is theoretically indicated but in whom it is contraindicated or not is not clinically appropriate) or secondary to a chronic kidney disease, and parathyroid carcinomas.
In pediatric patients, data on its use are restricted due to its recent marketing authorization (2017) and limited to dialysis patients suffering from secondary hyperPTH.
Nevertheless, some patients with phosphocalcic pathologies without renal insufficiency must be treated off-label by cinacalcet in the presence of severe hyperPTH, without any other chronic treatment available to date.
The objective of this study is therefore to evaluate the use in France of cinacalcet in phosphocalcic pathologies without renal insufficiency, in order to obtain efficacy and safety data in order to improve our knowledge on the management of these orphan diseases.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Cinacalcet | Patients with primary or secondary hyperPTH resulting from phosphocalcic pathology treated by cinacalcet |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Serum PTH concentrations results | Other | To compare serum PTH concentrations results in patients with primary or secondary hyperPTH resulting from phosphocalcic pathology before and three months after initiation of cinacalcet therapy. |
| Measure | Description | Time Frame |
|---|---|---|
| Serum PTH concentration | 3 months after treatment initiation |
| Measure | Description | Time Frame |
|---|---|---|
| Serum PTH concentration | 1 month after treatment initiation | |
| Serum PTH concentration | 6 months after treatment initiation | |
| Serum PTH concentration |
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Inclusion Criteria:
Exclusion Criteria:
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Patients with phosphocalcic diseases suffering from hyperPTH without end-stage renal failure.
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Justine BACCHETTA, MD | Contact | 4 27 85 61 30 | +33 | justine.bacchetta@chu-lyon.fr |
| Sacha FLAMMIER, PHD | Contact | 4 27 85 66 69 | +33 | sacha.flammier@chu-lyon.fr |
| Name | Affiliation | Role |
|---|---|---|
| Justine BACCHETTA, MD | Service de Néphrologie, Rhumatologie et Dermatologie Pédiatriques | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Endocrinologie Diabète et Maladies Métaboliques - Hôpital Gabriel Montpied | Clermont-Ferrand | France |
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| ID | Term |
|---|---|
| D006962 | Hyperparathyroidism, Secondary |
| D049950 | Hyperparathyroidism, Primary |
| D006961 | Hyperparathyroidism |
| ID | Term |
|---|---|
| D010279 | Parathyroid Diseases |
| D004700 | Endocrine System Diseases |
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| 12 months after treatment initiation |
| Serum PTH concentration | 3 years after treatment initiation |
| Service de Néphrologie Pédiatrique -Hôpital Jeanne de Flandre | Lille | 59037 | France |
|
| Service d'Endocrinologie Pédiatrique - Hôpital de la mère et de l'Enfant | Limoges | 87042 | France |
|
| Pole Femme Mère Enfant - Pédiatrie spécialisée - Centre Hospitalier Universitaire | Montpellier | 34090 | France |
|
| Service de Néphrologie pédiatrique - Clinique Médicale Pédiatrique | Nantes | 44093 | France |
|
| Service d'endocrinologie et Diabétologie Pédiatrique-Hôpital Robert Debré | Paris | 75019 | France |
|
| Service de Diabétologie et endocrinologie pédiatriques - Centre Hospitalier Universitaire | Reims | 51100 | France |
|
| Pôle Néphrologie-Urologie-Diabétologie-Endocrinologie | Strasbourg | 67091 | France |
|
| Service d'Endocrinologie, Maladies Osseuses, Gynécologie, Génétique | Toulouse | 31059 | France |
|
| Unité Endocrinologie, Nutrition, Diabétologie -Hôpital Bretonneau | Tours | 37044 | France |
|